Longitudinal Trends in Health-Related Quality of Life in Adults With Cystic Fibrosis

Dill, Edward J.; Dawson, Ree; Sellers, Deborah E.; Robinson, Walter M.; Sawicki, Gregory S.

doi:10.1378/chest.12-1404

Cited by 49 publications

(84 citation statements)

References 25 publications

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“…40 In the present study, there was no evidence of association between BMI and any of the WHOQOL-BREF domain scores, although BMI was a predictor of CFQOL scores. Similar results were demonstrated by Dill et al 41 in a longitudinal study with subject-reported outcomes: nutritional status was associated with CFQ-Revised physical domain scores. Our study demonstrated a significant effect for sex as a predictor of CFQOL scores for the weight and body image domains, but there were no associations with any WHOQOL-BREF scores.…”

Section: Discussionsupporting

confidence: 88%

Relationship Between Clinical Variables and Health-Related Quality of Life in Young Adult Subjects With Cystic Fibrosis

Forte

Perin

et al. 2015

Respir Care

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BACKGROUND: Health-related quality of life (HRQOL) has received much attention in patients with cystic fibrosis (CF). The goal of this study was to evaluate the association between clinical, lung function, sleep quality, and polysomnographic variables with 2 HRQOL questionnaires, the shorterversion World Health Organization Quality of Life (WHOQOL-BREF) and Cystic Fibrosis Quality of Life (CFQOL) questionnaires, in adult subjects with CF. METHODS: In a cross-sectional study, 51 subjects underwent clinical evaluation and overnight polysomnography and answered WHO-QOL-BREF, CFQOL, Pittsburgh Sleep Quality Index, and Epworth Sleepiness Scale questionnaires. In addition, pulmonary function tests, 6-min walk tests, and echocardiography were performed. RESULTS: For WHOQOL-BREF scores, the sleep quality index was associated with the physical domain; the percent-of-predicted 6-min walk distance (6MWD) and sleepiness scale were associated with the psychological domain; the percent-of-predicted FEV 1 and sleep quality index were associated with the social relationship domain; and the sleep quality index was associated with the environment domain. For CFQOL scores, age at diagnosis, clinical score, and sleep quality index were associated with the physical functioning domain; the percent-of-predicted 6MWD and pulmonary arterial systolic pressure were associated with the role domain; sex and sleep quality index were associated with the vitality domain; the apnea-hypopnea index was associated with the emotional functioning domain; sex and body mass index (BMI) were associated with the body image domain; the percent-of-predicted 6MWD and sleep quality index were associated with the health perception domain; age, sex, BMI, and arousal index were associated with the weight domain; age, sex, percent-of-predicted FEV 1 , percent-of-predicted 6MWD, and pulmonary arterial systolic pressure were associated with the respiratory symptom domain; and the clinical score was associated with the digestive symptom domain. CONCLUSIONS: The sleep quality index score, 6MWD, sleepiness scale score, and FEV 1 were predictors of WHOQOL-BREF scores. Age at diagnosis, clinical score, sleep quality score, 6MWD, sex, apnea-hypopnea index, BMI, current age, arousal index, FEV 1 , and pulmonary arterial systolic pressure were predictors of CFQOL scores.

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Section: Discussionsupporting

confidence: 88%

Relationship Between Clinical Variables and Health-Related Quality of Life in Young Adult Subjects With Cystic Fibrosis

Forte

Perin

et al. 2015

Respir Care

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“…S4 in the supplemental material). We employed the CFQ-R because it is a generally accepted framework for discussing health-related quality of life for this disease (79)(80)(81). Whether the ebb and flow of respiratory symptoms in CF are associated with changes in microbial gene transcription is not yet known.…”

Section: Discussionmentioning

confidence: 99%

Use of a Multiplex Transcript Method for Analysis of Pseudomonas aeruginosa Gene Expression Profiles in the Cystic Fibrosis Lung

et al. 2016

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The discovery of therapies that modulate Pseudomonas aeruginosa virulence or that can eradicate chronic P. aeruginosa lung infections associated with cystic fibrosis (CF) will be advanced by an improved understanding of P. aeruginosa behavior in vivo. We demonstrate the use of multiplexed Nanostring technology to monitor relative abundances of P. aeruginosa transcripts across clinical isolates, in serial samples, and for the purposes of comparing microbial physiology in vitro and in vivo. The expression of 75 transcripts encoded by genes implicated in CF lung disease was measured in a variety of P. aeruginosa strains as well as RNA serial sputum samples from four P. aeruginosa-colonized subjects with CF collected over 6 months. We present data on reproducibility, the results from different methods of normalization, and demonstrate high concordance between transcript relative abundance data obtained by Nanostring or transcriptome sequencing (RNA-Seq) analysis. Furthermore, we address considerations regarding sequence variation between strains during probe design. Analysis of P. aeruginosa grown in vitro identified transcripts that correlated with the different phenotypes commonly observed in CF clinical isolates. P. aeruginosa transcript profiles in RNA from CF sputum indicated alginate production in vivo, and transcripts involved in quorum-sensing regulation were less abundant in sputum than strains grown in the laboratory. P. aeruginosa gene expression patterns from sputum clustered closely together relative to patterns for laboratory-grown cultures; in contrast, laboratory-grown P. aeruginosa showed much greater transcriptional variation with only loose clustering of strains with different phenotypes. The clustering within and between subjects was surprising in light of differences in inhaled antibiotic and respiratory symptoms, suggesting that the pathways represented by these 75 transcripts are stable in chronic CF P. aeruginosa lung infections. Several factors complicate the comparison of in vitro studies on Pseudomonas aeruginosa with infections in the cystic fibrosis (CF) lung. First, many different P. aeruginosa strains cause CF lung infections; thus, reliance on commonly used laboratory strains might limit our understanding of P. aeruginosa in CF (1-3). Second, laboratory or cell culture studies rarely incorporate coinfecting species and lack components of the lung environment, such as immune response factors, that shape P. aeruginosa phenotypes (4, 5). We know little about how these environmental stimuli influence P. aeruginosa (6-8). To complicate matters further, a single CF sputum sample contains mixtures of P. aeruginosa genotypes and phenotypes. A recent study has suggested that a mixture of strains influenced traits such as drug response in ways that were difficult to predict from the study of single strains (9). Clinical isolates of P. aeruginosa, even from the same respiratory sample, can have striking differences in phenotypes, including colony morphology, quorum-sensing regulation, and motili...

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“…Most CF patients 6 years or older, the age group in whom the test is performed routinely in the clinic, are familiar with the test, and with appropriate training accurate measurements are easy to obtain. FEV 1 is considered an appropriate surrogate outcome for CF studies since low FEV 1 values are strongly associated with increased mortality, and decreased quality of life [5][6][7]. 3.…”

Section: Fevmentioning

confidence: 99%

“…Moving forward, it would be preferential to provide data for both absolute and relative changes in FEV 1 for all clinical trials to facilitate the comparison of effect sizes among studies. 6. FEV 1 across the age range Spirometry while easy to perform does require that subjects actively cooperate during testing to ensure both the inspiration to total lung capacity and the forced exhalation reflect their maximal effort.…”

Section: Lack Of Standardized Reportingmentioning

confidence: 99%

Physiologic endpoints for clinical studies for cystic fibrosis

Stanojevic¹,

Ratjen²

2016

Journal of Cystic Fibrosis

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The cystic fibrosis (CF) drug development pipeline promises many exciting new treatments for patients with CF, all which will require clinical studies to prove their benefits on CF lung disease. Historically many pivotal CF studies have used the Forced Expiratory Volume in 1s (FEV1) as the primary outcome measure, and after demonstrating significant improvements in the treatment group relative to placebo have led to regulatory approval of therapies for routine clinical care. Widespread implementation of these therapies has subsequently led to significant improvements in outcomes for patients with CF. While preserving lung function has obvious benefits to CF patients, as more patients maintain FEV1 in the normal range, it has become increasingly difficult to conduct clinical trials using FEV1 as the primary outcome measure. With multiple concurrent trials competing to enroll from the same pool of patients, there is a need for novel approaches to study end points as well as new physiological outcomes for CF therapeutic trials. In this review we will discuss some of the limitations of FEV1 in the current era of CF care, describe alternative physiological endpoints and outline areas for further research.

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Longitudinal Trends in Health-Related Quality of Life in Adults With Cystic Fibrosis

Cited by 49 publications

References 25 publications

Relationship Between Clinical Variables and Health-Related Quality of Life in Young Adult Subjects With Cystic Fibrosis

Relationship Between Clinical Variables and Health-Related Quality of Life in Young Adult Subjects With Cystic Fibrosis

Use of a Multiplex Transcript Method for Analysis of Pseudomonas aeruginosa Gene Expression Profiles in the Cystic Fibrosis Lung

Physiologic endpoints for clinical studies for cystic fibrosis

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